New muscular dystrophy cooperative research center established at UNC

The University of North Carolina at Chapel Hill has been awarded a five-year, $7 million grant to establish a muscular dystrophy research center named in honor of the late Sen. Paul D. Wellstone of Minnesota, who earned his undergraduate and doctoral degrees at UNC.

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The new Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center at UNC joins a list of six existing Wellstone Centers that have been established by the National Institutes of Health. Wellstone was an advocate for muscular dystrophy issues in Congress before he was killed in a plane crash in 2002.

According to the National Institute for Neurological Disorders and Stroke, muscular dystrophy is a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms are seen in infancy or childhood, while others may not appear until middle age or later.

R. Jude Samulski, Ph.D., professor of pharmacology in the School of Medicine, member of the Lineberger Comprehensive Cancer Center and director of the UNC Gene Therapy Center, will serve as director of UNC’s Wellstone Center. William Powers, M.D., Houston Merritt Professor and chair of neurology, will serve as co-director.

“A major theme of UNC’s Wellstone Center will be to develop novel, gene-based therapies for muscle disorders in the laboratory and then advance those therapies into the clinical setting,” Samulski said. Samulski is currently leading the nation’s first clinical trial of a gene therapy for Duchenne muscular dystrophy, which was launched in 2006.

Gene therapy is an experimental technique that uses genes to treat or prevent disease by inserting a gene into a patient’s cells instead of using drugs or surgery. It is currently only being tested for the treatment of diseases that have no other cures.

Samulski and colleagues at UNC have created a program comprised of experienced clinical investigators, expert basic laboratories and pre-clinical testing models focused on developing, testing and establishing therapeutic treatments for Duchenne muscular dystrophy and other genetic muscle disorders.

“The impetus for this effort stems from the fact that the UNC Gene Therapy Center has made significant strides in recent years to build the foundation that will enable development of a full-scale, bench-to-bedside effort bringing gene therapy for Duchenne muscular dystrophy closer to a reality,” Powers said.

A primary focus of the UNC Wellstone Center is to establish a clear understanding of the safety and efficacy of gene-based therapies that show potential for treatment and advancing these efforts into selected human clinical trials.

The center will pursue three projects. The first – headed by Powers along with Jon Wolff, M.D., of the University of Wisconsin, as co-principal investigator – is a Phase I clinical trial that will establish and ensure the safety of gene delivery to muscles affected by muscular dystrophy. The second project – headed by Xiao Xiao, Ph.D., distinguished professor of gene therapy in the UNC Eshelman School of Pharmacy, in collaboration with Joe Kornegay, D.V.M., Ph.D., professor of pathology and laboratory medicine in the School of Medicine – will investigate and improve methods of delivering gene-based therapies to large muscle groups. The third project, headed by Samulski, will optimize the best “delivery vehicle” for targeting and treating muscle disorders with gene-based therapies.

UNC’s Wellstone Center is one of two centers of excellence for muscular dystrophy research being newly funded by the NIH; the other is at Boston Biomedical Research Institute (BBRI).

The other six existing Wellstone Centers are at the University of Washington, the University of Rochester, the University of Pittsburgh, the University of Iowa, Children’s National Medical Center in Washington, D.C., and a joint center run by the University of Pennsylvania and Johns Hopkins University.

The centers are supported by cooperative agreements with three NIH institutes: the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Child Health and Human Development, and the National Institute of Neurological Disorders and Stroke. The Muscular Dystrophy Association has provided additional funds to expand the research at these centers.

The Wellstone Centers promote side-by-side basic, translational and clinical research and provide resources that can be used by muscular dystrophy researchers nationwide. They also serve as focal points for research collaborations, training and communication.

Wellstone attended UNC on a wrestling scholarship, graduating with a degree in political science in 1965 before going on to earn a Ph.D. in political science in 1969. Wellstone then accepted a job as a professor of political science at Carleton College in Northfield, Minn., where he taught until his election to the Senate in 1990. He served in the Senate from 1991 until his untimely death.